Regenerative medicine has not yet proved itself in the clinic beyond rare diseases or conditions of limited public health importance. With recent scientific discoveries opening up new approaches to regenerative medicine, the challenge is to use these to extend the regenerative approach to major diseases and conditions.

Projects should focus on innovative translational research to develop regenerative processes towards the ultimate clinical goal of addressing unmet clinical needs of large patient groups. Proposals should be based on new approaches such as genome editing or gene therapy, transdifferentiation or in vivo reprogramming, cell therapy and transplantation, 3D bioprinting, organoids or use of combined products (non-exhaustive list for illustrative purposes only).

In all cases, proposals should explain in what way their approach is regenerative. Research on improved methods of tissue and organ transplantation is included on the condition that there is a clear regenerative step in the process.

The project may focus on any step(s) on the innovation chain, from early testing and characterization of regenerative mechanisms to preclinical research, proof of concept or clinical trial. Sex and gender differences should be investigated, where relevant. Projects should include a section on the proposed therapy’s exploitation potential, regulatory and commercialisation strategy and how it would be made available and delivered to patients.

• Potential new regenerative therapies to address unmet clinical needs of large patient groups identified.
• Europe’s position in translational regenerative medicine strengthened.
• New therapies for major human diseases and conditions, and new approaches for therapy taken further in the development pipeline.

NGS can provide insights on a person’s genetic susceptibility to disease, diagnostic information, and predictive indications about treatment outcome. It also allows to embrace simultaneously different molecular pathways of disease evolution and to identify actionable mutations in a patient for medical decision and further research. In addition, it requires less sample material than multiple tests and therefore reduces risk and inconvenience for patients.

However, the introduction of NGS in clinical practice is hampered by its cost, the availability of proper NGS tests, and diagnostic errors resulting from insufficient quality assurance, technological bias and complex interpretation of data.

The objective is to implement NGS in routine diagnostics for personalised medicine and scale up demand-driven innovation for healthcare systems. This includes organisational, economical, technical and clinical aspects.

It should lead to NGS tests, clinically validated procedures (including sex analysis), quality assurance schemes, tools and methods for data collection, management, analysis and interpretation, with a view to assist clinical decision-making and foster medical research and innovation.

Transferability and cloud based NGS data analyses should be considered, as appropriate. Input from initiatives like the EJP Cofund on rare diseases and ERNs should be considered when relevant. Ethical issues should be addressed.

For grants awarded under this topic for Pre-Commercial procurement it is expected that results could contribute to European or international standards. Therefore, the respective option of Article 28.2 of the Model Grant Agreement will be applied.

• New NGS platforms and use of NGS tests in routine diagnostics for personalised medicine.
• Accepted new European standards and quality assurance schemes with respect to NGS.
• Strengthening of implementation of personalised medicine and improved clinical decisions and health outcomes for the benefits of patients.
• Contribution to the sustainability of healthcare systems.
• Growth and benefit to the European industry, in particular SMEs.